Smit Y, Verweij L, Currie A, Janssen J J W M, Posthuma E F M, Dekker A, Hermens R P M G, Blijlevens N M A
Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands.
Department of Internal Medicine, Reinier de Graaf Hospital, Delft, the Netherlands.
Cancer Med. 2025 Apr;14(8):e70880. doi: 10.1002/cam4.70880.
Toxicity monitoring should be modernized to include real-world patient-reported data. However, little is known about how stakeholders view the incorporation of real-world patient-reported toxicity symptoms into guidelines. This gap hinders the development of a sustained learning healthcare environment and limits the incorporation of this data into daily care.
This qualitative study, reported according to COREQ, involved interviews with 29 plus 10 chronic myeloid leukemia (CML) patients and 18 CML clinicians, including eight hematologists/guideline developers. The interviews were audio-recorded, transcribed, and independently coded in Atlas.ti. A framework, adapted from systematically sourced literature, was used for coding. Codes were assessed as either beneficial or limiting. An expert panel of all CML guideline developers completed and prioritized the identified knowledge gaps through a RAND-modified Delphi procedure.
Thirty-one benefits and limitations of systematically monitoring patient-reported toxicity symptoms in the real world were identified. Compared to an existing framework, novel benefits centered around the use of aggregated data: Participants viewed real-world patient-reported toxicity symptoms as a way to systematically include patients' toxicity symptoms in the guidelines; personalize guideline advice; and fill knowledge gaps. The expert panel agreed on 14 knowledge gaps in chronic myeloid leukemia care that could be addressed through such data. Novel limitations focused on the suitability, acceptance, and applicability of toxicity symptom monitoring in routine clinical practice. Participants felt that this monitoring does not establish a causal link between medication and symptoms, and it has no added value over open conversation.
The benefits and limitations of adopting patient-reported real-world toxicity symptom monitoring need to be leveraged and addressed to ensure maximum value and uptake. Guideline developers viewed aggregated data as beneficial. The identified knowledge gaps provide concrete points of action for CML guideline development.
毒性监测应实现现代化，纳入真实世界中患者报告的数据。然而，对于利益相关者如何看待将真实世界中患者报告的毒性症状纳入指南，我们知之甚少。这一差距阻碍了持续学习型医疗环境的发展，并限制了此类数据在日常护理中的应用。
本定性研究按照COREQ报告，对29名加10名慢性髓性白血病（CML）患者以及18名CML临床医生进行了访谈，其中包括8名血液学家/指南制定者。访谈进行了录音、转录，并在Atlas.ti中进行独立编码。采用从系统获取的文献改编的框架进行编码。编码被评估为有益或有局限性。所有CML指南制定者组成的专家小组通过兰德修改的德尔菲程序完成并对识别出的知识差距进行了优先排序。
确定了在现实世界中系统监测患者报告的毒性症状的31项益处和局限性。与现有框架相比，新的益处集中在汇总数据的使用上：参与者将真实世界中患者报告的毒性症状视为将患者的毒性症状系统纳入指南的一种方式；使指南建议个性化；以及填补知识空白。专家小组就慢性髓性白血病护理中可通过此类数据解决的14个知识差距达成了共识。新的局限性集中在毒性症状监测在常规临床实践中的适用性、可接受性和实用性。参与者认为这种监测没有建立药物与症状之间的因果联系，并且与开放式对话相比没有附加价值。
需要利用并解决采用患者报告的真实世界毒性症状监测的益处和局限性，以确保实现最大价值并得到应用。指南制定者认为汇总数据是有益的。识别出的知识差距为CML指南的制定提供了具体的行动要点。
